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Alnylam Pharmaceuticals Initiates RNAi Therapeutic Development Program for Respiratory Syncytial Virus Infection

Posted on: Tuesday, 4 January 2005, 09:00 CST

CAMBRIDGE, Mass., Jan. 4 /PRNewswire-FirstCall/ -- Alnylam Pharmaceuticals, Inc. , a leading RNAi therapeutics company, today announced it has initiated a therapeutic development program for the treatment of respiratory syncytial virus (RSV) infection. RSV is a major cause of respiratory illness in young children, the elderly and immune- deficient patient populations. RSV infects nearly every child by the age of two years and typically results in cold-like symptoms; however, it may lead to lower respiratory tract infections or respiratory failure. Children born prematurely or with pulmonary, cardiac and immune deficiencies are at the highest risk for severe RSV infection that can result in hospitalization, morbidity and even death. Alnylam will implement its Direct RNAi(TM) technology to treat RSV infection by targeting key viral genes at the site of infection.

"We are excited to initiate this new program in RSV disease, as it further advances our leadership in the development of Direct RNAi therapeutics for important diseases with unmet medical needs," said John Maraganore, President and Chief Executive Officer of Alnylam Pharmaceuticals. "We plan to move forward aggressively to develop a novel treatment for RSV infection, present additional in vivo data at upcoming scientific meetings and initiate clinical trials in the first half of 2006."

This program is based in part on research published last week in Nature Medicine by Professor Sailen Barik and his colleagues from the University of South Alabama, College of Medicine. This research demonstrated that RSV infection could be prevented and treated with high potency in an animal model by intranasal administration of short interfering RNAs (siRNAs), the molecules that induce RNAi.

"Our published results showing in vivo inhibition of RSV through intranasal delivery of siRNAs clearly highlight the power of harnessing the natural process of RNAi," said Sailen Barik, Ph.D., Professor in the Department of Biochemistry at the University of South Alabama, College of Medicine. "We are excited to be working with Alnylam Pharmaceuticals, the leading RNAi therapeutics company, as we believe they are best able to rapidly advance our scientific breakthrough to positively impact the significant RSV patient population worldwide."

The RSV therapeutic program is the second therapeutic development program announced by Alnylam, the first being the age-related macular degeneration program Alnylam is conducting in collaboration with Merck. Both programs aim to develop Direct RNAi products, which are RNAi therapeutics that will be administered directly at sites of diseases, such as the lung, eye or the brain. In addition, Alnylam has a number of earlier-stage pre-clinical programs including a program in Parkinson's disease and additional programs as part of its collaborations with Merck.

In connection with its new RSV program, Alnylam has completed a licensing agreement with the University of South Alabama that provides Alnylam with an exclusive license to certain intellectual property stemming from the work of Dr. Sailen Barik. The license covers RNAi compositions and methods of treating pulmonary viral infections with siRNAs.

About RSV

RSV is a highly contagious virus that causes infections in both the upper and lower respiratory tract. RSV infects nearly every child by the age of two years and is a major cause of hospitalization due to respiratory infection in infants born prematurely, children with lung or congenital heart disease, the elderly, and other adult immune-compromised populations. RSV infection typically results in cold-like symptoms but can lead to more serious respiratory illness such as croup, pneumonia and bronchiolitis, and in extreme cases severe morbidity and death. RSV is responsible for up to an estimated 125,000 pediatric hospitalizations each year in the United States. As a result, there is a significant need for novel therapeutics to treat patients who become infected with RSV.

About RNAi

RNA interference, or RNAi, is a naturally occurring mechanism within cells for selectively silencing and regulating specific genes that is potentially the basis for a new class of therapeutic products. Since many diseases are caused by the inappropriate activity of specific genes, the ability to silence and regulate such genes selectively through RNAi could provide a means to treat a wide range of human diseases. The discovery of RNAi has been heralded by many as a major breakthrough, and the journal Science named RNAi the top scientific achievement of 2002, as well as one of the top 10 scientific advances of 2003.

About Alnylam

Alnylam is a biopharmaceutical company seeking to develop and commercialize novel therapeutics based on RNA interference, or RNAi. Growing from its foundation as the world's first company focused on RNAi therapeutics, the company's leadership in the field of RNAi is supported by its preeminent founders and advisors and its strengths in fundamental patents, technology, and know-how that underlie the commercialization of RNAi therapeutics. Alnylam is developing a pipeline of RNAi products using Direct RNAi(TM) to treat ocular, central nervous system, and respiratory diseases and Systemic RNAi(TM) to treat a broad range of diseases, including oncologic, metabolic, and autoimmune diseases. The company's global headquarters are in Cambridge, Massachusetts. For additional information, please visit http://www.alnylam.com/.

About the University of South Alabama, College of Medicine

The University of South Alabama (USA), College of Medicine is a vibrant and expanding center providing all facets of medical education, research and patient care. Supported by the University of South Alabama Health System, the USA College of Medicine faculty enjoys a reputation of excellence in teaching, research and service. The college has received national attention for its leading edge facilities and research. The USA College of Medicine is located in Mobile, Alabama. For additional information, please visit http://www.southalabama.edu/com.

Alnylam Pharmaceuticals Forward-Looking Statements

Various statements in this release concerning our future expectations, plans, prospects and future operating results constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including risks related to: our approach to discover and develop novel drugs, which is unproven and may never lead to marketable products; our ability to obtain additional funding to support our business activities; our dependence on third parties for development, manufacture, marketing, sales and distribution of our products; the successful development of products, all of which are in early stages of development; obtaining regulatory approval for products; competition from others using technology similar to ours and others developing products for similar uses; obtaining, maintaining and protecting intellectual property utilized by our products; and our short operating history; as well as those risks more fully discussed in the "Certain Factors That May Affect Future Results" section of our most recent Form 10-Q filed with the Securities and Exchange Commission. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We do not assume any obligation to update any forward- looking statements.

Alnylam Pharmaceuticals, Inc

CONTACT: Investor Contact, Lilian Stern of Stern Investor Relations,Inc., +1-212-362-1200, or Media Contact, Kathryn Morris of KMorrisPR,+1-845-635-9828, both for Alnylam Pharmaceuticals, Inc.; or Holly W. Meadowsof University of South Alabama College of Medicine Office of TechnologyDevelopment, +1-251-460-7932

Web site: http://www.alnylam.com/http://www.southalabama.edu/com

Source: PRNewswire-FirstCall

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